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In Utero Gene Editing Technique Developed At CMU And Yale Could Be Used In Humans

Carnegie Mellon University
Carnegie Mellon University Professor Danith Ly, who was part of the research team to study curing genetic diseases in utero.

A study by Carnegie Mellon University and Yale University has found a gene editing technique administered in fetal mice can cure a genetic blood disease before birth. This is the first time this technique has been successful, and it could be tested in humans within a year.

Scientists used healthy donor DNA to alter the genetic code of a mouse carrying fetuses with mutation that causes beta thalassemia. The disease reduces the production of hemoglobin and hampers the delivery of oxygen to tissues.

The genetic disorder was effectively cured in the fetuses with a single treatment, and no side effects were detected.

The Centers for Disease Control estimates that each year, three percent of babies are born with genetic defects. CMU professor Danith Ly said many of these disorders can be detected during pregnancy.

"There are a lot of these genetic diseases that we can diagnose but we have no way of correcting them before a child is born," he said.

Right now, there are two options for parents who discover a serious genetic defect in a fetus -- either terminate the pregnancy or go forward knowing the child could live a short and difficult life.

"What we're doing is we're trying to provide a third option," Ly said. 

The success of this procedure in mice doesn't guarantee it will work for humans -- Ly said lots of medical techniques successful in mice don't work for people. But, he said a startup company pending launch is looking to move this technology to preliminary clinical trials probably within a year. If approved by the FDA, Ly said this technology could be available as a treatment option in three to six years.